Tehran University of Medical Sciences

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Faculty Members have conducted research related to the Viral-Based Gene Therapy Clinical Trials for Immune Deficiencies and Blood Disorders From 2013 Until 2023 - an Overview
Maryam Behfar
Maryam Behfar

Associate Professor of Pediatric Hematology and Oncology

Department Pediatrics

School of Medicine

Pediatric Cell and Gene Therapy Research Center

Tehran University of Medical Sciences

All Documents
1. Dyskeratosis Congenita Future: Hematopoietic Stem Cell Transplantation or Gene Therapy?, Gene Reports (2025)
2. Gut Microbiota Variation and Diversity and Gut Graft-Versus-Host Disease (Gvhd) in Pediatrics: A Systematic Review, Transplant Immunology (2025)
3. Hematopoietic Stem Cell Transplantation for C1q Deficiency: A Study on Behalf of the Ebmt Inborn Errors Working Party, Journal of Clinical Immunology (2025)
4. Successful of Autologous Hematopoietic Stem Cell Mobilization With Plerixafor Combined With G-Csf in Pediatric Neuroblastoma Patients, a Single Center Experience, Transfusion and Apheresis Science (2025)
5. The Tale of Pediatric Hematopoietic Stem Cell Transplantation in Iran: An Uphill Battle, Journal of Pediatric Hematology/Oncology (2025)
6. Viral-Based Gene Therapy Clinical Trials for Immune Deficiencies and Blood Disorders From 2013 Until 2023 - an Overview, Regenerative Therapy (2025)
7. How Age, Sex and Transfusion Affects the Incidence of Endocrine and Bone Density Disorders in Major Thalassemic Patients, Iranian Journal of Public Health (2024)
8. Phase I Study of Safety and Efficacy of Allogeneic Natural Killer Cell Therapy in Relapsed/Refractory Neuroblastomas Post Autologous Hematopoietic Stem Cell Transplantation, Scientific Reports (2024)
9. Posterior Reversible Encephalopathy Syndrome in Pediatric Hematopoietic Stem Cell Transplantation With Beta Major Thalassemia: The Association Between the Pres Occurrence and Class of Beta Major Thalassemia, Clinical Transplantation (2024)
10. The Application of Umbilical Cord Blood-Derived Platelet Gel for Skin Ulcers Associated With Chronic Graft-Versus-Host Disease in Pediatrics: A Randomized Trial, Transplantation and Cellular Therapy (2024)
11. The Reconstitution of T-Cells After Allogeneic Hematopoietic Stem Cell Transplant in a Pediatric Patient With Congenital Amegakaryocytic Thrombocytopenia (Camt), Endocrine# Metabolic and Immune Disorders - Drug Targets (2024)
12. A Comprehensive Comparison Between Tbi Vs Non-Tbi-Based Conditioning Regimen in Pediatric Patients With Acute Lymphoblastic Leukemia: A Systematic Review and Meta-Analysis, Leukemia Research (2023)
13. Clinical and Immunological Characteristics of 69 Leukocyte Adhesion Deficiency-I Patients, Pediatric Allergy and Immunology (2023)
14. Clinical and Immunological Characteristics of 69 Leukocyte Adhesion Deficiency-I Patients, Pediatric Allergy and Immunology (2023)
15. Clinical, Immunological, and Genetic Findings in Iranian Patients With Mhc-Ii Deficiency: Confirmation of C.162Delg Rfxank Founder Mutation in the Iranian Population, Journal of Clinical Immunology (2023)
16. Clinical, Immunological, and Genetic Findings in Iranian Patients With Mhc-Ii Deficiency: Confirmation of C.162Delg Rfxank Founder Mutation in the Iranian Population, Journal of Clinical Immunology (2023)
17. Disturbance in the Reconstitution of Distinct T-Cell Subsets and the Incidence of Gvhd Following Allo-Hsct in Pediatric Patients With Non-Malignant Hematological Disorders, Immunology Letters (2023)
18. Effect of Early Bacillus Calmette-Guerin Vaccination of Pediatric Severe Combined Immunodeficiency Patients on the Outcome of Hematopoietic Stem Cell Transplantation Using a Reduced-Intensity Conditioning Regimen, Transplantation and Cellular Therapy (2023)
19. Evaluation of Safety and Efficacy of Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells in Pediatric Bronchiolitis Obliterans Syndrome (Bos) After Allogeneic Hematopoietic Stem Cell Transplantation (Allo-Hsct), Stem Cell Research and Therapy (2023)
20. Fanconi Anemia Phenotypic and Transplant Outcomes' Associations in Iranian Patients, Health Science Reports (2023)
21. Intra-Lesion Injection of Activated Natural Killer (Nk) Cells in Recurrent Malignant Brain Tumors, International Immunopharmacology (2023)
22. T Helper 17 and Regulatory T-Cell Profile and Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Patients With Beta-Thalassemia, Transplant Immunology (2023)
23. Total Body Irradiation-Free Haploidentical Peripheral Blood Stem Cell Transplantation Compared to Related and Unrelated Donor Transplantation in Pediatrics With Acute Lymphoblastic Leukemia, Pediatric Blood and Cancer (2023)
24. Unbalanced T-Cell Subsets in Pediatric Patients With Beta-Thalassemia, Human Immunology (2023)
25. Adoptive Nk-Cell Transfer As a Potential Treatment Paradigm for Wilms Tumor: A Preclinical Study, Pediatric Blood and Cancer (2022)
26. Escalated Dose Donor Lymphocyte Infusion Treatment in Patients With Primary Immune Deficiencies After Hsct With Reduced-Intensity Conditioning Regimen, Hematology/ Oncology and Stem Cell Therapy (2022)
27. Fludarabine-Based Reduced Intensity Conditioning for Allogeneic Hematopoietic Stem Cell Transplantation in a Pediatric Patient With Bone Marrow Failure Syndrome Type 3, Journal of Pediatric Hematology/Oncology (2022)
28. Long-Term Follow-Up of Patients With Lps-Responsive Beige-Like Anchor Protein Deficiency After Reduced-Intensity Conditioning for Allogeneic Hematopoietic Stem Cell Transplantation: Report of Two Cases, Hematology# Transfusion and Cell Therapy (2022)
29. Spontaneous Xenogeneic Gvhd in Wilms' Tumor Patient-Derived Xenograft Models and Potential Solutions, Animal Models and Experimental Medicine (2022)
30. Successful Allogeneic Stem Cell Transplantation With a Reduced-Intensity Conditioning in a Case of Leukocyte Adhesion Deficiency Type Iii, Hematology# Transfusion and Cell Therapy (2022)
31. Analysis of Determinant Factors of Liver Fibrosis Progression in Ex-Thalassemic Patients, International Journal of Hematology (2021)
32. Clinical Effects of Covid-19 on Hematopoietic Stem Cell Transplant Outcomes in Pediatric Patients, Experimental and Clinical Transplantation (2021)
33. Covid-19 in Transplant Recipient Children: An Iranian Referral Hospital-Based Study, Acta bio-medica : Atenei Parmensis (2021)
34. Covid-19 in Transplant Recipient Children: An Iranian Referral Hospital-Based Study, Acta Biomedica (2021)
35. Fludarabine-Based Reduced-Intensity Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Pediatric Patient With Il10 Receptor Deficiency, Pediatric Transplantation (2021)
36. Radiation-Free Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Adult Acute Lymphoblastic Leukemia: A Comparison of Outcomes Between Patients With and Without Central Nervous System Involvement, Leukemia Research (2021)
37. Relationship Between Endocrine Changes and Bone Markers in Pediatric Thalassemic Patients After Hematopoietic Stem Cell Transplantation, Minerva Pediatrics (2021)
38. Successful Allogeneic Stem Cell Transplantation With Fludarabine-Based Reduced Intensity Conditioning in Bone Marrow Failure Syndrome 4, Pediatric Transplantation (2021)
39. Posterior Reversible Encephalopathy Syndrome After Allogeneic Stem Cell Transplantation in Pediatric Patients With Fanconi Anemia, a Prospective Study, Biology of Blood and Marrow Transplantation (2020)
40. A Single-Center Experience With Hematopoietic Stem Cell Transplantation for Pediatric Acute Lymphoblastic Leukemia: A Modest Pitch for Non-Total Body Irradiation Conditioning Regimens, Experimental and Clinical Transplantation (2019)
41. Outcome of Allogeneic Hematopoietic Stem Cell Transplantation on Diamond-Blackfan Anemia Using Busulfan-Based Myeloablative Regimen, Turkish Journal of Pediatrics (2019)
42. Impact of Hematopoietic Stem Cell Transplant on Vep and Abr Values of the Patients With Malignant Infantile Osteopetrosis, European Journal of Paediatric Neurology (2018)
43. Long-Term Safety of Short-Term Administration of Filgrastim (Rhg-Csf) and Leukophresis Procedure in Healthy Children: Application of Peripheral Blood Stem Cell Collection in Pediatric Donors, Biology of Blood and Marrow Transplantation (2018)
44. Short-Term Assessment of Hsct Effects on the Hypothalamus-Pituitary Axis in Pediatric Thalassemic Patients, Archives of Iranian Medicine (2018)
45. First Report of Pediatric Hematopoietic Stem Cell Transplantation Activities in the Eastern Mediterranean Region From 1984 to 2011: On Behalf of the Pediatric Cancer Working Committee of the Eastern Mediterranean Blood and Marrow Transplantation Group, Bone Marrow Transplantation (2017)
46. Natural Killer Cells From the Subcutaneous Adipose Tissue Underexpress the Nkp30 and Nkp44 in Obese Persons and Are Less Active Against Major Histocompatibility Complex Class I Non-Expressing Neoplastic Cells, Frontiers in Immunology (2017)
47. Nk Cell-Derived Exosomes From Nk Cells Previously Exposed to Neuroblastoma Cells Augment the Antitumor Activity of Cytokine-Activated Nk Cells, Journal of Immunotherapy (2017)
48. Non-Sibling Hematopoietic Stem Cell Transplantation Using Myeloablative Conditioning Regimen in Children With Maroteaux-Lamy Syndrome: A Brief Report, Pediatric Transplantation (2017)
49. Outcome Analysis of Pediatric Patients With Acute Lymphoblastic Leukemia Treated With Total Body Irradiation–Free Allogeneic Hematopoietic Stem Cell Transplantation: Comparison of Patients With and Without Central Nervous System Involvement, Biology of Blood and Marrow Transplantation (2017)
50. Long-Term Outcomes of Fludarabine, Melphalan and Antithymocyte Globulin As Reduced-Intensity Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Children With Primary Immunodeficiency Disorders: A Prospective Single Center Study, Bone Marrow Transplantation (2016)
51. Peripheral Blood Versus Bone Marrow Transplant in Patients With Aplastic Anemia, an Unresolved Issue, Bone Marrow Transplantation (2016)
52. Comparison of Bone Mineral Density Changes in Pediatric Thalassemic Patients With and Without Hematopoietic Stem Cell Transplant, Experimental and Clinical Transplantation (2015)
53. Efficacy of Hepatic T2∗ Mri Values and Serum Ferritin Concentration in Predicting Thalassemia Major Classification for Hematopoietic Stem Cell Transplantation, Pediatric Transplantation (2015)
54. Efficiency of Allogeneic Hematopoietic Sct From Hla Fully-Matched Non-Sibling Relatives: A New Prospect of Exploiting Extended Family Search, Bone Marrow Transplantation (2015)
55. Hematopoietic Sct in Iranian Children 1991-2012, Bone Marrow Transplantation (2015)
56. Matched Related Donor Hematopoietic Stem Cell Transplantation Results in a Long-Term Follow-Up of a Pediatric Acquired Severe Aplastic Anemia Subset: A Stem Cell Source Perspective, Pediatric Transplantation (2015)
57. Non-Total Body Irradiation Myeloablative Conditioning With Intravenous Busulfan and Cyclophosphamide in Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis, Pediatric Transplantation (2015)
58. Radiologic Resolution of Malignant Infantile Osteopetrosis Skeletal Changes Following Hematopoietic Stem Cell Transplantation, Pediatric Blood and Cancer (2015)